Retinitis Pigmentosa Treatment Market

Retinitis Pigmentosa Treatment Market

Global retinitis pigmentosa treatment market reached US$ YY million in 2023 and is expected to reach US$ YY million by 2031, growing at a CAGR of YY% during the forecast period 2024-2031.

Retinitis pigmentosa (RP) is a group of inherited diseases that damage the light-sensitive rods and cones in the retina, the back part of the eyes. The rods, which provide side (peripheral) and night vision are affected more than the cones, which provide color and clear central vision. RP is the most common type of inherited eye disease. People with RP experience a gradual decline in their vision, because photoreceptors degenerate.

A lot of advances have been made in recent years in the field of RP including the introduction of gene therapy. Using low vision aids and assistive devices. There are a range of magnifiers and technology that can identify things or people that the wearer points to. Using sunglasses and other methods to avoid exposure to too much light. Light may make RP worse. Treating associated conditions, such as cystoid macular edema (CME), which may happen with RP. The FDA has approved voretigene neparvovec-ryzl (Luxturna), a gene therapy product to treat a specific type of retinitis pigmentosa. People with mutations in both copies of the RP65 gene may benefit from this type of therapy.

Market Dynamics: Drivers

Increasing demand for innovative therapeutics

The increasing demand for innovative therapeutics is expected to drive the market over the forecast period. Retinitis Pigmentosa is a rare genetic disorder with very limited treatment options. The lack of effective therapies for the condition creates a substantial unmet medical need, expected to increase the demand for innovative solutions that can address the causes of the disease and provide meaningful benefits to patients. The major market players are performing clinical trials to develop innovative therapeutics.

For instance, on September 13, 2023, Ocugen Inc. announced a clinical study update for retinitis pigmentosa (RP) participants treated in the Phase 1/2 trial to assess the safety and efficacy of OCU400 for RP associated with NR2E3 and Rhodopsin (RHO) mutations and Leber congenital amaurosis (LCA) with mutation(s) in the CEP290 gene.

Innovative therapeutics, such as gene therapies and advanced treatments, have the potential to offer improved patient outcomes compared to other traditional or symptomatic approaches. Patients and healthcare providers often seek treatments that can not only manage symptoms but also slow down or halt the progression of the disease. So, there is an increasing demand for the innovative gene therapies.

For instance, on March 30, 2023, Nanoscope Therapeutics Inc., released the topline results from the Phase 2b multicenter, randomized, double-masked, sham-controlled RESTORE clinical trial (NCT04945772) of MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy for vision restoration in advanced retinitis pigmentosa (RP), irrespective of gene mutation. MCO-010 has received both orphan drugs and fast-track designations from the U.S. Food and Drug Administration (FDA).

There is an increasing prevalence of retinitis pigmentosa globally, which increases the demand for advanced innovative therapeutics for better treatment. For instance, according to the National Organization for Rare Diseases, RP affects about 1 in 3,000 to 1 in 4,000 people in the world. This means that with a population of about 330 million in the United States in 2021, about 82,500 to 110,000 people in the United States have RP. With a worldwide population presently estimated at over 7.74 billion, it can be estimated that approximately 1.94 to 2.58 million people around the world have these disorders. This increases the demand for innovative treatment approaches.

For instance, on April 06, 2023, Endogena Therapeutics concluded the dose-escalation stage of its Phase l/lla trial of EA-2353 to treat retinitis pigmentosa (RP). Based on the positive safety and tolerability results, the trial will include patients for the expansion cohort, where the highest dose will be given to patients to find the possible efficacy of EA-2353. Altogether 14 patients with RP as a result of any pathologic genetic mutation are being enrolled at around six US sites.

Further, the increasing prevalence of retinitis pigmentosa, rising research activities and clinical trials to develop more advanced therapies, increasing demand for gene therapies, rising aging population, rising awareness about the condition and increasing advancements in the development of novel therapies are the factors expected to drive the market over the forecast period.

Restraints

Factors such as complications and side effects associated with the various therapies, the high cost of the treatment, availability of alternative treatment options, genetic complexity and heterogeneity, limited understanding of disease mechanisms and long clinical development timelines are the factors expected to hamper the market.

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Segment Analysis

The global retinitis pigmentosa treatment market is segmented based on disease type, treatment type, end-user and region.

The gene therapy segment accounted for approximately 36.8% of the retinitis pigmentosa treatment market share

The gene therapy segment is expected to hold the largest market share over the forecast period. Gene therapy for retinitis pigmentosa involves introducing functional copies of a defective gene into the patient's cells to compensate for the genetic mutation causing the condition. For instance, on August 22, 2023, ViGeneron GmbH cleared the European Medicines Agency (EMA) approval for a Clinical Trial Application (CTA) of its new gene therapy, VG901. VG901 uses an adeno-associated virus (AAV) vector to treat CNGA1-associated retinitis pigmentosa (RP).

Voretigene neparvovec-ryzl (Luxturna) is a gene therapy product approved by the FDA to treat a specific type of retinitis pigmentosa with mutations in both copies of the RP65 gene. Luxturna delivers a functional copy of the RPE65 gene to the retinal cells, aiming to restore vision.

Besides Luxturna, various other gene therapies for retinitis pigmentosa are under investigation in clinical trials. These trials are exploring different gene therapy approaches, including the use of various viral vectors to deliver therapeutic genes, to address various genetic mutations associated with the condition. The major market players are focusing on the development of gene therapies for the management of the retinitis pigmentosa.

For instance, on June 13, 2023, Beacon Therapeutics launched new generation of gene therapies for a range of retinal diseases. Beacon’s lead clinical asset is AGTC-501, a gene therapy program in phase 2 clinical trials for the treatment of X-linked retinitis pigmentosa (XLRP). The therapy, which was part of Syncona’s acquisition of AGTC in 2022, addresses the photoreceptor damage caused by XLRP by expressing the full-length RPGR protein.

In addition, on September 7, 2023, Opus Genetics dosed the first patient in its Phase 1/2 gene therapy clinical for Leber congenital amaurosis 5 (LCA5), which causes significant vision loss in children. The Phase 1/2 clinical trial, enrolling nine adult patients, is being conducted at the University of Pennsylvania. Once safety in adults has been established and confirmed by the US Food and Drug Administration, Opus plans to dose pediatric patients.

Geographical Analysis

North America accounted for approximately 41.6% of the market share

North America region is expected to hold the largest market share over the forecast period owing to the strong presence of major players and increasing research activities for developing advanced therapies. North America especially the United States is a known hub for its strong presence of major players such as pharmaceutical companies, which actively focus on the development of novel therapies for better treatment.

For instance, on June 28, 2022, MeiraGTx Holdings plc released the top-line data from the Phase 1/2 clinical study MGT009 (NCT03252847) of botaretigene sparoparvovec (formerly referred to as AAV-RPGR), an investigational gene therapy in development for the treatment of patients with X-linked retinitis pigmentosa (XLRP) with disease-causing variants in the RPGR gene.

In addition, there are increasing research activities and clinical trials in the region for developing more advanced therapeutics for the better management of retinitis pigmentosa, which also helps to drive the market in the region. As the research activities and clinical trials increase, the therapeutics are developed more precisely by reducing the complications associated with the different types of therapies, resulting in better patient outcomes.

For instance, on October 25, 2023, Dyno Therapeutics, Inc. launched its Dyno eCap 1 capsid product, a leading eye AAV gene delivery vector with best-in-class potential. The Dyno eCap 1 vector provides significantly improved delivery to the eye compared to other externally engineered capsids, including transduction throughout multiple layers of the retina. Enabling effective delivery to all cells in the eye, and in particular across the entire retina, is crucial to enable gene therapies to treat retinitis pigmentosa.

Competitive Landscape

The major global players in the retinitis pigmentosa treatment market include Spark Therapeutics, Inc., jCyte, Inc., Coave Therapeutics, Kiora Pharmaceuticals, Inc., Aldeyra Therapeutics, Inc., Beacon Therapeutics, ViGeneron GmbH, Nanoscope Therapeutics Inc., SparingVision, Inc. and Endogena Therapeutics Inc. among others.

COVID-19 Impact Analysis

The COVID-19 pandemic significantly impacted the global retinitis pigmentosa treatment market. Some studies analyzed common biological pathways genes shared by retinitis pigmentosa and COVID-19 by newest bioinformatics tools, trying to unveil new biological mechanisms that might involve the SARS-CoV-2 and to identify new biomarkers useful to predict the increased susceptibility towards SARS-CoV-2. In addition, during the pandemic, the clinical trials for retinitis pigmentosa were temporarily disrupted due to a redirected focus towards COVID-19 and related restrictions.

Market Segmentation

By Disease Type
• Non-Syndromic Retinitis Pigmentosa
Autosomal Dominant
Autosomal Recessive
X-Linked
• Syndromic Retinitis Pigmentosa

By Treatment Type
• Gene Therapy
• Low Vision Aids and Assistive Devices
• Sunglasses
• Vitamin A Palmitate
• Omega-3 Fatty Acids
• Lutein Plus Zeaxanthin
• Surgery
• Others

By End-User
• Hospitals
• Specialty Clinics
• Ophthalmic Centers
• Ambulatory Surgical Centers
• Others

By Region
• North America
U.S.
Canada
Mexico
• Europe
Germany
U.K.
France
Spain
Italy
Rest of Europe
• South America
Brazil
Argentina
Rest of South America
• Asia-Pacific
China
India
Japan
Australia
Rest of Asia-Pacific
• Middle East and Africa

Why Purchase the Report?
• To visualize the global retinitis pigmentosa treatment market segmentation based on disease type, treatment type, end-user and region as well as understand key commercial assets and players.
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The global retinitis pigmentosa treatment market report would provide approximately 61 tables, 63 figures and 187 Pages.

Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies